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Current Therapy of the Patients with MDS: Walking towards Personalized Therapy
Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8153316/ https://www.ncbi.nlm.nih.gov/pubmed/34068316 http://dx.doi.org/10.3390/jcm10102107 |
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author | Palacios-Berraquero, Maria Luisa Alfonso-Piérola, Ana |
author_facet | Palacios-Berraquero, Maria Luisa Alfonso-Piérola, Ana |
author_sort | Palacios-Berraquero, Maria Luisa |
collection | PubMed |
description | Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and to increase the quality of life. Currently, available drugs for newly diagnosed low-risk MDS include growth factor support, lenalidomide and immunosuppressive therapy. Additionally, luspatercept has recently been added to treat patients with MDS with ring sideroblasts, who are not candidates or have lost the response to erythropoiesis-stimulating agents. Treatment of high-risk patients is aimed to improve survival. To date, the only currently approved treatments are hypomethylating agents and allogeneic stem cell transplantation. However, the future for MDS patients is promising. In recent years, we are witnessing the emergence of multiple treatment combinations based on hypomethylating agents (pevonedistat, magrolimab, eprenetapopt, venetoclax) that have proven to be effective in MDS, even those with high-risk factors. Furthermore, the approval in the US of an oral hypomethylating agent opens the door to exclusively oral combinations for these patients and their consequent impact on the quality of life of these patients. Relapsed and refractory patients remain an unmet clinical need. We need more drugs and clinical trials for this profile of patients who have a dismal prognosis. |
format | Online Article Text |
id | pubmed-8153316 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-81533162021-05-27 Current Therapy of the Patients with MDS: Walking towards Personalized Therapy Palacios-Berraquero, Maria Luisa Alfonso-Piérola, Ana J Clin Med Review Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and to increase the quality of life. Currently, available drugs for newly diagnosed low-risk MDS include growth factor support, lenalidomide and immunosuppressive therapy. Additionally, luspatercept has recently been added to treat patients with MDS with ring sideroblasts, who are not candidates or have lost the response to erythropoiesis-stimulating agents. Treatment of high-risk patients is aimed to improve survival. To date, the only currently approved treatments are hypomethylating agents and allogeneic stem cell transplantation. However, the future for MDS patients is promising. In recent years, we are witnessing the emergence of multiple treatment combinations based on hypomethylating agents (pevonedistat, magrolimab, eprenetapopt, venetoclax) that have proven to be effective in MDS, even those with high-risk factors. Furthermore, the approval in the US of an oral hypomethylating agent opens the door to exclusively oral combinations for these patients and their consequent impact on the quality of life of these patients. Relapsed and refractory patients remain an unmet clinical need. We need more drugs and clinical trials for this profile of patients who have a dismal prognosis. MDPI 2021-05-13 /pmc/articles/PMC8153316/ /pubmed/34068316 http://dx.doi.org/10.3390/jcm10102107 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Palacios-Berraquero, Maria Luisa Alfonso-Piérola, Ana Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title_full | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title_fullStr | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title_full_unstemmed | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title_short | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
title_sort | current therapy of the patients with mds: walking towards personalized therapy |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8153316/ https://www.ncbi.nlm.nih.gov/pubmed/34068316 http://dx.doi.org/10.3390/jcm10102107 |
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